CL2021000669A1 - Rnai agents to inhibit the expression of 17beta-hsd type 13- (hsd17b13), their compositions and methods of use. - Google Patents

Rnai agents to inhibit the expression of 17beta-hsd type 13- (hsd17b13), their compositions and methods of use.

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Publication number
CL2021000669A1
CL2021000669A1 CL2021000669A CL2021000669A CL2021000669A1 CL 2021000669 A1 CL2021000669 A1 CL 2021000669A1 CL 2021000669 A CL2021000669 A CL 2021000669A CL 2021000669 A CL2021000669 A CL 2021000669A CL 2021000669 A1 CL2021000669 A1 CL 2021000669A1
Authority
CL
Chile
Prior art keywords
hsd17b13
17beta
inhibit
expression
compositions
Prior art date
Application number
CL2021000669A
Other languages
Spanish (es)
Inventor
Rui Zhu
Zhen Li
Shawn A Morales
Original Assignee
Arrowhead Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Arrowhead Pharmaceuticals Inc filed Critical Arrowhead Pharmaceuticals Inc
Publication of CL2021000669A1 publication Critical patent/CL2021000669A1/en

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1137—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against enzymes
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/7088—Compounds having three or more nucleosides or nucleotides
    • A61K31/713—Double-stranded nucleic acids or oligonucleotides
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/50—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates
    • A61K47/51—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent
    • A61K47/54—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient the non-active ingredient being chemically bound to the active ingredient, e.g. polymer-drug conjugates the non-active ingredient being a modifying agent the modifying agent being an organic compound
    • A61K47/549—Sugars, nucleosides, nucleotides or nucleic acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P1/00—Drugs for disorders of the alimentary tract or the digestive system
    • A61P1/16—Drugs for disorders of the alimentary tract or the digestive system for liver or gallbladder disorders, e.g. hepatoprotective agents, cholagogues, litholytics
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12Y—ENZYMES
    • C12Y101/00—Oxidoreductases acting on the CH-OH group of donors (1.1)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2300/00—Mixtures or combinations of active ingredients, wherein at least one active ingredient is fully defined in groups A61K31/00 - A61K41/00
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/14—Type of nucleic acid interfering N.A.
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
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    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
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    • C12N2310/00—Structure or type of the nucleic acid
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    • C12N2310/323—Chemical structure of the sugar modified ring structure
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/33—Chemical structure of the base
    • C12N2310/331—Universal or degenerate base
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/343—Spatial arrangement of the modifications having patterns, e.g. ==--==--==--
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
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    • C12N2310/3521—Methyl
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    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/353—Nature of the modification linked to the nucleic acid via an atom other than carbon
    • C12N2310/3533—Halogen
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    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/31—Combination therapy

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  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
CL2021000669A 2018-09-19 2021-03-18 Rnai agents to inhibit the expression of 17beta-hsd type 13- (hsd17b13), their compositions and methods of use. CL2021000669A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862733320P 2018-09-19 2018-09-19
US201862773707P 2018-11-30 2018-11-30
US201962890220P 2019-08-22 2019-08-22

Publications (1)

Publication Number Publication Date
CL2021000669A1 true CL2021000669A1 (en) 2021-10-29

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CL2021000669A CL2021000669A1 (en) 2018-09-19 2021-03-18 Rnai agents to inhibit the expression of 17beta-hsd type 13- (hsd17b13), their compositions and methods of use.

Country Status (20)

Country Link
US (1) US20220056454A1 (en)
EP (1) EP3852769A4 (en)
JP (1) JP7488254B2 (en)
KR (1) KR20210061380A (en)
CN (1) CN113164509A (en)
AU (1) AU2019342117A1 (en)
BR (1) BR112021005137A2 (en)
CA (1) CA3109553A1 (en)
CL (1) CL2021000669A1 (en)
CO (1) CO2021003446A2 (en)
CR (1) CR20210186A (en)
EC (1) ECSP21027262A (en)
IL (1) IL281597A (en)
MX (1) MX2021003077A (en)
PE (1) PE20211212A1 (en)
PH (1) PH12021550423A1 (en)
SG (1) SG11202101698WA (en)
TW (1) TW202024324A (en)
UY (1) UY38382A (en)
WO (1) WO2020061177A1 (en)

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BR112019004178A2 (en) * 2016-09-02 2019-05-28 Arrowhead Pharmaceuticals Inc bleach binders
AU2019239971A1 (en) * 2018-03-21 2020-09-03 Regeneron Pharmaceuticals, Inc. 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof
JOP20210158A1 (en) 2018-12-21 2023-01-30 Ionis Pharmaceuticals Inc Modulators of hsd17b13 expression
CN116234907A (en) * 2020-06-01 2023-06-06 美国安进公司 RNAI constructs for inhibiting HSD17B13 expression and methods of use thereof
WO2022098748A1 (en) * 2020-11-06 2022-05-12 Inipharm, Inc. Uses for hsd17b13 inhibitors
JP2024502282A (en) 2020-12-23 2024-01-18 リジェネロン・ファーマシューティカルズ・インコーポレイテッド Treatment of liver disease with cell death-inducing DFFA-like effector B (CIDEB) inhibitors
CA3216332A1 (en) * 2021-04-22 2022-10-27 Tuojie Biotech (Shanghai) Co., Ltd. Sirna targeting 17b-hydroxysteroid dehydrogenase type 13 and sirna conjugate
WO2023039076A1 (en) * 2021-09-08 2023-03-16 Aligos Therapeutics, Inc. Modified short interfering nucleic acid (sina) molecules and uses thereof
TW202340466A (en) * 2021-12-16 2023-10-16 大陸商上海拓界生物醫藥科技有限公司 Dsrna, preparation method and application thereof
US20230193392A1 (en) 2021-12-20 2023-06-22 Regeneron Pharmaceuticals, Inc. Methods Of Identifying And Evaluating Liver Inflammation And Liver Fibrosis In A Subject By Determining A Stratified Score Based On Gene Expression
WO2023138663A1 (en) * 2022-01-20 2023-07-27 上海拓界生物医药科技有限公司 Dsrna, use thereof and preparation method therefor
WO2023196941A1 (en) 2022-04-08 2023-10-12 Arrowhead Pharmaceuticals, Inc. Treatment of a non-alcoholic fatty liver disease
WO2023212019A1 (en) * 2022-04-28 2023-11-02 Enanta Pharmaceuticals, Inc. Pyrimidinone-containing 17-beta-hydroxysteroid dehydrogenase type 13 inhibitors
WO2023208109A1 (en) * 2022-04-29 2023-11-02 北京福元医药股份有限公司 Sirna for inhibiting hsd17b13 expression, conjugate and pharmaceutical composition thereof, and use thereof
TW202400791A (en) * 2022-05-06 2024-01-01 大陸商蘇州瑞博生物技術股份有限公司 Nucleic acid, composition and conjugate comprising same, preparation method therefor, and use thereof

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AU1067697A (en) * 1995-12-05 1997-06-27 Jouko Antero Oikarinen Hsd17b1 promoter, enhancer, silencer and use thereof
AU2005248147A1 (en) 2004-05-11 2005-12-08 Alphagen Co., Ltd. Polynucleotides for causing RNA interference and method for inhibiting gene expression using the same
CN103520724B (en) * 2013-10-23 2016-05-25 江苏美迪森生物医药有限公司 The new purposes of the inhibitor of HSD17B13 albumen or its encoding gene
WO2016081444A1 (en) * 2014-11-17 2016-05-26 Alnylam Pharmaceuticals, Inc. Apolipoprotein c3 (apoc3) irna compositions and methods of use thereof
JOP20210043A1 (en) * 2015-10-01 2017-06-16 Arrowhead Pharmaceuticals Inc Compositions and Methods for Inhibiting Gene Expression of LPA
AU2017229469B2 (en) 2016-03-07 2023-02-23 Arrowhead Pharmaceuticals, Inc. Targeting ligands for therapeutic compounds
TWI826365B (en) * 2017-01-10 2023-12-21 美商愛羅海德製藥公司 ALPHA-1 ANTITRYPSIN (AAT) RNAi AGENTS, COMPOSITIONS INCLUDING AAT RNAi AGENTS, AND METHODS OF USE
RU2021133626A (en) * 2017-01-23 2022-01-31 Регенерон Фармасьютикалз, Инк. HSD17B13 OPTIONS AND THEIR APPLICATIONS
AU2019239971A1 (en) * 2018-03-21 2020-09-03 Regeneron Pharmaceuticals, Inc. 17beta-hydroxysteroid dehydrogenase type 13 (HSD17b13) iRNA compositions and methods of use thereof

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CN113164509A (en) 2021-07-23
CO2021003446A2 (en) 2021-04-08
SG11202101698WA (en) 2021-04-29
KR20210061380A (en) 2021-05-27
CR20210186A (en) 2021-06-14
AU2019342117A2 (en) 2021-03-11
CA3109553A1 (en) 2020-03-26
UY38382A (en) 2020-03-31
MX2021003077A (en) 2021-05-27
PE20211212A1 (en) 2021-07-05
IL281597A (en) 2021-05-31
EP3852769A1 (en) 2021-07-28
US20220056454A1 (en) 2022-02-24
WO2020061177A1 (en) 2020-03-26
JP2022501040A (en) 2022-01-06
TW202024324A (en) 2020-07-01
JP7488254B2 (en) 2024-05-21
EP3852769A4 (en) 2023-12-13
ECSP21027262A (en) 2021-05-31
AU2019342117A1 (en) 2021-03-04
BR112021005137A2 (en) 2021-06-15
PH12021550423A1 (en) 2021-09-20

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